BR112022020423A2 - COMPOUNDS AND METHODS FOR SPLICING MODULATION - Google Patents
COMPOUNDS AND METHODS FOR SPLICING MODULATIONInfo
- Publication number
- BR112022020423A2 BR112022020423A2 BR112022020423A BR112022020423A BR112022020423A2 BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2 BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2
- Authority
- BR
- Brazil
- Prior art keywords
- compounds
- methods
- splicing
- splicing modulation
- modulation
- Prior art date
Links
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/50—Pyridazines; Hydrogenated pyridazines
- A61K31/502—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with carbocyclic ring systems, e.g. cinnoline, phthalazine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/50—Pyridazines; Hydrogenated pyridazines
- A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/517—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
- C07D401/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings
- C07D401/12—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
- C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
- C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
- C07D403/04—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
- C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
- C07D413/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
- C07D417/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
- C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
- C07D471/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
- C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
- C07D471/08—Bridged systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Neurosurgery (AREA)
- Urology & Nephrology (AREA)
- Pulmonology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Diabetes (AREA)
- Biomedical Technology (AREA)
- Obesity (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Saccharide Compounds (AREA)
- Plural Heterocyclic Compounds (AREA)
- Replacement Of Web Rolls (AREA)
Abstract
COMPOSTOS E MÉTODOS PARA MODULAÇÃO DE SPLICING. A presente invenção refere-se a compostos e composições relacionadas que, entre outros, modulam o splicing de ácido nucleico, por exemplo, splicing de um pré-mRNA, assim como a métodos de uso dos mesmos.COMPOUNDS AND METHODS FOR SPLICING MODULATION. The present invention relates to compounds and related compositions which, inter alia, modulate nucleic acid splicing, for example, splicing of a pre-mRNA, as well as methods of using the same.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063007331P | 2020-04-08 | 2020-04-08 | |
US202063044318P | 2020-06-25 | 2020-06-25 | |
US202063072922P | 2020-08-31 | 2020-08-31 | |
US202063126494P | 2020-12-16 | 2020-12-16 | |
PCT/US2021/026481 WO2021207554A1 (en) | 2020-04-08 | 2021-04-08 | Compounds and methods for modulating splicing |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112022020423A2 true BR112022020423A2 (en) | 2022-11-29 |
Family
ID=75690721
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022020423A BR112022020423A2 (en) | 2020-04-08 | 2021-04-08 | COMPOUNDS AND METHODS FOR SPLICING MODULATION |
Country Status (14)
Country | Link |
---|---|
US (2) | US20230159496A1 (en) |
EP (1) | EP4132646A1 (en) |
JP (1) | JP2023520916A (en) |
KR (1) | KR20220167298A (en) |
CN (1) | CN115551593A (en) |
AU (1) | AU2021253587A1 (en) |
BR (1) | BR112022020423A2 (en) |
CA (1) | CA3174353A1 (en) |
CL (1) | CL2022002780A1 (en) |
CR (1) | CR20220566A (en) |
IL (1) | IL297137A (en) |
MX (1) | MX2022012678A (en) |
TW (1) | TW202208344A (en) |
WO (2) | WO2021207554A1 (en) |
Families Citing this family (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
RU2020105929A (en) | 2017-08-04 | 2021-09-06 | Скайхоук Терапьютикс, Инк. | METHODS AND COMPOSITIONS FOR MODULATING SPLICING |
TW202208358A (en) | 2020-05-13 | 2022-03-01 | 美商Chdi基金會股份有限公司 | Htt modulators for treating huntington’s disease |
CA3233973A1 (en) * | 2021-10-13 | 2023-04-20 | Dominic Reynolds | Compounds and methods for modulating nucleic acid splicing |
CA3239110A1 (en) * | 2021-11-24 | 2023-06-01 | Dominic Reynolds | Compounds and methods for modulating splicing |
WO2023133225A1 (en) * | 2022-01-05 | 2023-07-13 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
US11957759B1 (en) | 2022-09-07 | 2024-04-16 | Arvinas Operations, Inc. | Rapidly accelerated fibrosarcoma (RAF) degrading compounds and associated methods of use |
Family Cites Families (23)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DK1163237T3 (en) * | 1999-03-17 | 2004-08-02 | Astrazeneca Ab | amide derivatives |
GB0324790D0 (en) * | 2003-10-24 | 2003-11-26 | Astrazeneca Ab | Amide derivatives |
TWI320783B (en) * | 2005-04-14 | 2010-02-21 | Otsuka Pharma Co Ltd | Heterocyclic compound |
BRPI0817061A2 (en) * | 2007-09-12 | 2015-03-24 | Wyeth Llc | Pyrrolidinylalkylisoquinolinone and -isoindolinoline derivatives as histamine-3 antagonists |
EP3406252B1 (en) | 2012-02-10 | 2020-05-13 | PTC Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
US8729263B2 (en) | 2012-08-13 | 2014-05-20 | Novartis Ag | 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions |
WO2014151147A1 (en) * | 2013-03-15 | 2014-09-25 | Intellikine, Llc | Combination of kinase inhibitors and uses thereof |
CN107428729B (en) | 2015-02-09 | 2021-07-16 | 豪夫迈·罗氏有限公司 | Compounds for the treatment of cancer |
MA41898A (en) * | 2015-04-10 | 2018-02-13 | Hoffmann La Roche | BICYCLIC QUINAZOLINONE DERIVATIVES |
EP4249472A3 (en) | 2015-05-30 | 2023-12-13 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
RU2747644C2 (en) * | 2015-09-23 | 2021-05-11 | Янссен Фармацевтика Нв | Bigeteroaryl-substituted 1,4-benzodiazepines and ways of their use for cancer treatment |
MX2021001091A (en) | 2015-12-10 | 2022-04-26 | Ptc Therapeutics Inc | Methods for treatng huntington's disease. |
US10913730B2 (en) * | 2016-10-10 | 2021-02-09 | Dong-A Socio Holdings Co., Ltd. | Heteroaryl compounds and their use as Mer inhibitors |
EA201991309A1 (en) | 2016-11-28 | 2019-11-29 | RNA SPLICING MODULATION METHODS | |
JP6754505B2 (en) * | 2017-01-22 | 2020-09-09 | 福建広生堂薬業股▲ふん▼有限公司 | ASK1 inhibitor, its preparation method and use |
JP2020523365A (en) | 2017-06-14 | 2020-08-06 | ピーティーシー セラピューティクス,インコーポレーテッド | Method of modifying RNA splicing |
RU2020105929A (en) | 2017-08-04 | 2021-09-06 | Скайхоук Терапьютикс, Инк. | METHODS AND COMPOSITIONS FOR MODULATING SPLICING |
JP7195328B2 (en) | 2017-09-25 | 2022-12-23 | スカイホーク・セラピューティクス・インコーポレーテッド | Methods and compositions for screening and identifying splicing modulators |
CN109721597B (en) * | 2017-10-30 | 2021-05-14 | 上海迪诺医药科技有限公司 | Pyridoazepine derivative, and pharmaceutical composition and application thereof |
US11098059B2 (en) * | 2017-11-08 | 2021-08-24 | Merck Sharp & Dohme Corp. | PRMT5 inhibitors |
MA51200A (en) * | 2017-12-02 | 2020-10-07 | Galapagos Nv | NEW COMPOUNDS AND ASSOCIATED PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT OF DISEASES |
US11530207B2 (en) | 2018-04-10 | 2022-12-20 | Skyhawk Therapeutics, Inc. | Compounds for the treatment of cancer |
WO2020004594A1 (en) | 2018-06-27 | 2020-01-02 | 株式会社リボルナバイオサイエンス | Prophylactic or therapeutic agent for spinal muscular atrophy |
-
2021
- 2021-04-08 MX MX2022012678A patent/MX2022012678A/en unknown
- 2021-04-08 CN CN202180027107.3A patent/CN115551593A/en active Pending
- 2021-04-08 BR BR112022020423A patent/BR112022020423A2/en unknown
- 2021-04-08 IL IL297137A patent/IL297137A/en unknown
- 2021-04-08 AU AU2021253587A patent/AU2021253587A1/en active Pending
- 2021-04-08 EP EP21722078.9A patent/EP4132646A1/en active Pending
- 2021-04-08 CR CR20220566A patent/CR20220566A/en unknown
- 2021-04-08 KR KR1020227038394A patent/KR20220167298A/en active Search and Examination
- 2021-04-08 US US17/917,729 patent/US20230159496A1/en active Pending
- 2021-04-08 WO PCT/US2021/026481 patent/WO2021207554A1/en active Application Filing
- 2021-04-08 TW TW110112826A patent/TW202208344A/en unknown
- 2021-04-08 JP JP2022561080A patent/JP2023520916A/en active Pending
- 2021-04-08 WO PCT/US2021/026437 patent/WO2021207532A1/en active Application Filing
- 2021-04-08 US US17/917,734 patent/US20230167093A1/en active Pending
- 2021-04-08 CA CA3174353A patent/CA3174353A1/en active Pending
-
2022
- 2022-10-07 CL CL2022002780A patent/CL2022002780A1/en unknown
Also Published As
Publication number | Publication date |
---|---|
MX2022012678A (en) | 2023-01-11 |
KR20220167298A (en) | 2022-12-20 |
EP4132646A1 (en) | 2023-02-15 |
AU2021253587A1 (en) | 2022-12-01 |
WO2021207532A1 (en) | 2021-10-14 |
US20230167093A1 (en) | 2023-06-01 |
WO2021207554A1 (en) | 2021-10-14 |
CL2022002780A1 (en) | 2023-04-28 |
IL297137A (en) | 2022-12-01 |
CN115551593A (en) | 2022-12-30 |
US20230159496A1 (en) | 2023-05-25 |
CA3174353A1 (en) | 2021-10-14 |
JP2023520916A (en) | 2023-05-22 |
CR20220566A (en) | 2023-01-23 |
TW202208344A (en) | 2022-03-01 |
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